Raptor Pharmaceutical Completes DR Cysteamine Phase 2b Clinical Trial in Cystinosis

By Raptor Pharmaceutical Corp., PRNE
Sunday, November 22, 2009

Goal of Trial Achieved Demonstrating Potentially Less Frequent Administration Using Lower Daily Dose

NOVATO, California, November 23 - Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP),
today announced results from a Phase 2b clinical trial of its proprietary
delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with
nephropathic cystinosis ("cystinosis"). The trial, conducted at the
University of California, San Diego ("UCSD") General Clinical Research
Center, evaluated safety, tolerability, pharmacokinetics and pharmacodynamics
of a single dose of DR Cysteamine in nine cystinosis patients.

(Logo: www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO )

    Phase 2b Clinical Trial Highlights:

    - DR Cysteamine demonstrated improved tolerability and the potential to
      reduce total daily dosage and administration frequency compared
      to immediate-release cysteamine bitartrate.

    - Pharmacokinetic evaluation showed that DR Cysteamine had a terminal
      half-life more than three times longer than the terminal half-life
      of immediate-release cysteamine bitartrate capsules.

    - Twice-daily DR Cysteamine may achieve the same pharmacodynamic result
      while using a daily dose 30% lower than immediate-release cysteamine
      bitartrate capsules administered four times daily.

    - No adverse events recorded during the clinical trial were determined by
      the principal investigator to be possibly or probably related to DR
      Cysteamine. Nine adverse events recorded in the clinical trial were
      determined to be possibly or probably related to immediate-release
      cysteamine bitartrate.

Raptor plans to meet with the Food and Drug Administration ("FDA") and
European Medicines Agency ("EMEA") to discuss the results of its phase 2b
study and its plans for a repeat-dose, pivotal, phase 3 clinical trial in
cystinosis patients. Upon receiving FDA and EMEA concurrence, Raptor intends
to initiate its phase 3 clinical trial at multiple sites in the US and Europe
in the first quarter of 2010.

DR Cysteamine is Raptor's proprietary, enteric-coated micro-bead
formulation of immediate release cysteamine bitartrate capsules, the current
standard of care for treating cystinosis.

Bruce Barshop, M.D., Ph.D., Professor of Clinical Pediatrics at UCSD and
principal investigator for the cystinosis clinical trial of DR Cysteamine,
stated, "The excellent tolerability of DR Cysteamine, as demonstrated in this
study, represents a major step forward in potentially improving the treatment
of cystinosis. Raptor's formulation has the potential to improve dosing
compliance and long-term outcomes for cystinosis patients."

Raptor's phase 2b clinical trial followed earlier clinical trials with an
enteric-coated cysteamine prototype conducted by Ranjan Dohil, M.D.,
Associate Professor of Pediatrics at UCSD and funded by the Cystinosis
Research Foundation ("CRF"). The CRF also supported Raptor's phase 2b
clinical trial.

Patrice P. Rioux, M.D., Ph.D., chief medical officer of Raptor, said,
"The results from our phase 2b trial are consistent with previous clinical
findings from Dr. Dohil and support our plans to provide a twice-daily
cysteamine therapy for cystinosis patients. We look forward to continuing our
development program of DR Cysteamine in cystinosis with a phase 3 clinical
trial."

Cystinosis is an inborn metabolic error characterized by the abnormal
transport of cystine, an amino acid, out of the lysosomes. Failure to treat
cystinosis can cause serious health consequences, including renal failure and
resultant kidney transplant, growth failure, rickets, photophobia and
blindness. Symptom onset typically occurs within the first year of life, when
cystine crystals accumulate in various tissues and organs, including the
kidneys, brain, liver, thyroid, pancreas, muscles and eyes.

About DR Cysteamine

DR Cysteamine is an enteric-coated micro-bead formulation of cysteamine
bitartrate. Raptor obtained an exclusive, worldwide license to DR Cysteamine,
as well as orphan drug designation from the FDA for DR Cysteamine for the
potential treatment of nephropathic cystinosis, through its December 2007
acquisition of Encode Pharmaceuticals. In March 2008, Raptor acquired an
exclusive worldwide license to intellectual property from the University of
California, San Diego
("UCSD") covering use of cysteamine and DR Cysteamine
for the potential treatment of non-alcoholic steatohepatitis ("NASH"), a
progressive liver disease believed to affect 2-5% of the U.S. population. In
October 2009, Raptor and UCSD announced positive interim data from the
six-month treatment phase of its Phase 2a clinical study of cysteamine
bitartrate in NASH patients. In November 2008 Raptor announced a
collaboration with Centre Hospitalier Universitaire d'Angers, in France, to
study DR Cysteamine in Huntington's Disease, a rare, genetic neurological
disease.

About Raptor Pharmaceutical Corp.

Raptor Pharmaceutical Corp. (Nasdaq: RPTP) ("Raptor") is dedicated to
speeding the delivery of new treatment options to patients by working to
improve existing therapeutics through the application of highly specialized
drug targeting platforms and formulation expertise. Raptor focuses on
underserved patient populations where it can have the greatest potential
impact. Raptor currently has product candidates in clinical development
designed to potentially treat nephropathic cystinosis, non-alcoholic
steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase
("ALDH2") deficiency, and a non-opioid solution designed to potentially treat
chronic pain.

Raptor's preclinical programs are based upon bioengineered novel drug
candidates and drug-targeting platforms derived from the human
receptor-associated protein ("RAP") and related proteins that are designed to
target cancer, neurodegenerative disorders and infectious diseases.

For additional information, please visit www.raptorpharma.com.

FORWARD LOOKING STATEMENTS

This document contains forward-looking statements as that term is defined
in the Private Securities Litigation Reform Act of 1995. These statements
relate to future events or our future results of operation or future
financial performance, including, but not limited to the following
statements: that Raptor will start a Phase 3 clinical trial in cystinosis
patients at multiple US and European sites in the first quarter of 2010, if
at all; that DR Cysteamine may allow for a 30% reduction in a patient's total
daily cysteamine dose compared to the current standard of care; that DR
Cysteamine can be administered in two doses per day instead of four, to
achieve the same pharmacodynamic result; that tolerability of DR Cysteamine,
as demonstrated in this study, potentially represents a major step forward in
the care of cystinosis patients; that Raptor's formulation could result in
greatly improved dosing compliance and better long-term outcomes for
cystinosis patients; and that any of Raptor's clinical and preclinical drug
candidates will result in approved therapeutics. These statements are only
predictions and involve known and unknown risks, uncertainties and other
factors, which may cause the Company's actual results to be materially
different from these forward-looking statements. Factors which may
significantly change or prevent the Company's forward looking statements from
fruition include: that Raptor may be unsuccessful at raising funds to
continue its development programs; Raptor may be unsuccessful in developing
any products or acquiring products; that Raptor's technology may not be
validated as it progresses further and its methods may not be accepted by the
scientific community; that Raptor is unable to retain or attract key
employees whose knowledge is essential to the development of its products;
that unforeseen scientific difficulties develop with the Company's process;
that Raptor's patents are not sufficient to protect essential aspects of its
technology; that competitors may invent better technology; and that Raptor's
products may not work as well as hoped or worse, that the Company's products
may harm recipients. As well, Raptor's products may never develop into useful
products and even if they do, they may not be approved for sale to the
public. Raptor cautions readers not to place undue reliance on any such
forward-looking statements, which speak only as of the date they were made.
Certain of these risks, uncertainties, and other factors are described in
greater detail in the Company's filings from time to time with the Securities
and Exchange Commission (the "SEC"), which Raptor strongly urges you to read
and consider, including Raptor's current report on Form 8-K as filed with the
SEC on November 17, 2009; the joint proxy statement/prospectus on Form S-4
filed with the SEC on August 19, 2009; Raptor's annual report on Form 10-K
filed with the SEC on March 27, 2009; and Raptor's quarterly report on Form
10-Q filed with the SEC on August 11, 2009, all of which are available free
of charge on the SEC's web site at www.sec.gov. Subsequent written and
oral forward-looking statements attributable to Raptor or to persons acting
on its behalf are expressly qualified in their entirety by the cautionary
statements set forth in Raptor's reports filed with the SEC. Raptor expressly
disclaims any intent or obligation to update any forward-looking statements.

    For more information, please contact:

    Karl Cahill, Investor Relations
    +1-858-531-6100
    kcahill@raptorpharma.com

    The Ruth Group
    Sara Ephraim Pellegrino (investors) / Janine McCargo (media)
    +1-646-536-7002 / +1-646-536-7033
    spellegrino@theruthgroup.com / jmccargo@theruthgroup.com

Karl Cahill, Investor Relations, +1-858-531-6100, kcahill at raptorpharma.com, Sara Ephraim Pellegrino (investors), +1-646-536-7002, spellegrino at theruthgroup.com, or Janine McCargo (media), +1-646-536-7033, jmccargo at theruthgroup.com, both of The Ruth Group

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